Welcome to….

The 7th Annual Meeting of

ISMPP

Anticipating Change in Medical

Publications:

Leading Now for the Future

International Society for Medical Publications Professionals

7th Annual Meeting

April 5, 2011

The Evolving Landscape for

Health Economics and Outcomes Research:

Implications for Medical Publications Professionals

Alan Lyles, ScD, MPH, RPh

Henry A. Rosenberg Professor of Public, Private and Nonprofit Partnerships,

University of Baltimore

Docent, University of Helsinki

AlanLyles@comcast.net

Why Are Health Economics &

Outcomes Research Needed?

(1) The evolution of comparative effectiveness

research & the Patient Centered Outcomes

Research Institute’s (PCORI's)

potential impact on medical publications,

(2) A vision for the future of medical publications in a

market-driven health care system, and

(3) Core competencies for medical publications

professionals in this new world.

3

4

“The right context

is worth 50 IQ points.”

- Alan Kay

Inventor of Object Oriented Programming

Visionary: Laptop Computer

5

National Health Accounts (2007)*

5* Source: World Health Organization (2010), http://www.who.int/nha/country/en/index.html

% USA China Russian

FederationFinland Germany Canada UK France

Life Expectancy

at Birth (2008)

78 74 68 80 80 81 80 81

Health

expenditures as %

GDP15.7 4.3 5.4 8.2 10.4 10.1 8.4 11.0

Government

expenditures as %

Health

expenditures

45.5 44.7 64.2 74.6 76.9 70.0 81.7 79.0

Private

expenditures as %

Health

expenditures

54.5 55.3 35.8 25.4 23.1 30.0 18.3 21.0

Government

health exp as %

All Government

expenditures

19.5 9.9 10.2 12.9 18.2 18.1 15.6 16.6

Notes: Percentages may not total 100% due to rounding.

Source: Kaiser Family Foundation calculations using NHE data from Centers for Medicare and Medicaid Services, Office of the Actuary, National Health

Statistics Group, at http://www.cms.hhs.gov/NationalHealthExpendData/ (see Historical; National Health Expenditures by type of service and source of

funds, CY 1960-2008; file nhe2008.zip).

Distribution of National Prescription Drug Expenditures by

Source of Payment, 1998-2008

Private Insurance

Consumer Out-of-Pocket

Payments

Public Funds

7

National Health Care Expenditure

Cost Components, United States, 1960-2005

Sources: K. Levit et al., Health Spending Rebound Continues in 2002 Health Affairs 2004;23(1):147-159.; Smith C, et al.

Health Spending Growth Slows in 2003. Health Affairs 2005;24(1):185-194; Smith C, et al. National Health Spending in

2003: Recent Slowdown Led By Prescription Drug Spending. Health Affairs 2006;25(1):186-196; Catlin A, et al. National

Health Spending in 2005: The Slowdown Continues. Health Affairs 2007;26(1):142-153.

Hospital Care

Physician

Services

Prescription Drugs

8

USA National Health Expenditures

& Sources of Funds 1966-2006SOURCE: Centers for Medicare & Medicaid Services, Office of the Actuary: Data from the National Health Statistics Group.

2006 1996 1986 1976 1966

Total ($USD B) 2,106 1,069 0.471 0.152 0.046

Per Capita ($USD) 7,026 3,938 1,932 687 230

Private (%) 54 54 59 58 70

Public (%) 46 46 41 41 30

Federal (%) 33 33 28 28 16

State & Local (%) 13 13 13 13 14

9

Persons Enrolled in HMOs, 1976 through 1997

Source 1: The data for 1976 through 1985 are for June of the year specified; those for 1986 through 1991 are for December. Data are

from Hoy et al and Group Health Association of America, with the permission of the publisher.

Source 2: New England Journal of Medicine, Sept 3, 1992, pp 744.; Health US 1999 Table 131

10

Source: IMS Health, 2001. Reported in PhRMA Industry Profile 2002. Generic share of countable units, such as tablets

Drugs expected to come off-patent 2005-2006:

http://www.gphaonline.org/AM/Template.cfm?Section=Resources1&CONTENTID=1597&TEMPLATE=/CM/HTMLDisplay.cfm

Generic Prescriptions as a

Percentage of the U S Pharmaceutical Market

11

Patients

Physicians

Pharmaceutical

Manufacturers

Figure 1. Evolution of Pharmaceutical Product Communications

and Prescribing Decisions

A. Before Rx Insurance

Patients

Physicians

Pharmaceutical

Manufacturers

B. After Managed Care

Pharmacy and

Therapeutics

Committee

Source: Lyles A. Promoting Pharmaceutical Products. Chapter 18 in Fulda TR and Wertheimer A, editors

of Pharmaceutical Public Policy . Haworth Press. ISBN: 978-0-7890-3059-7 2007

12

0

20

40

60

80

100

120

1979 1980 1981 1982 1983 1984 1985 1986 1987 1988 1989 1990 1991 1992 1993 1994 1995 1996

Education**

Device/Procedures*

Surgery

Pharmaceuticals

Number of Studies

Year

* Devices and procedures

** Education, behavior, other

Frequency of Economic Impact Studies

MEDTAPI n t e r n a t i o n a l

1313

Pharmacoeconomics1

“ … identifies, measures, and compares the

costs and consequences of

pharmaceutical products and services.”

1Bootman JL, Townsend RJ, McGhan WF, eds. Introduction to Pharmacoeconomics. In: Principles

of Pharmacoeconomics. 3rd ed. Cincinnati, Ohio. Harvey Whitney Books. 2004. ISBN

0929375270

*Distribution is statistically different from distribution for the previous year

shown (p<.05).

‡No statistical tests are conducted between 2003 and 2004 or between

2006 and 2007 due to the addition of a new category.

Note: Fourth-tier drug cost sharing information was not obtained prior to

2004.

Source: Kaiser/HRET Survey of Employer-Sponsored Health Benefits,

2000-2009.

Distribution of Covered Workers Facing Different Cost-Sharing Formulas

for Prescription Drug Benefits, 2000-2009: Patients’ Sticker Shock

‡

‡

15

Changing Patterns

of Pharmaceutical Innovation*

1989 – 2000: 1,035 New Drug Approvals by US FDA

• 65 % used active ingredients previously approved

• 35 % New Molecular Entities (NMEs)

• 58 % Standard Drugs (No significant clinical improvement)

• 42 % Priority drugs (Provide Clinical Improvement)

∑ = 153 (15%) used new active ingredients & represented significant clinical improvement*

National Institute of Health Care Management: http://www.nihcm.org/finalweb/innovations.pdf

16

1717

Pharmacoeconomics

(an Alternate Definition)

“ … a pseudodiscipline … conjured into

existence by the magic of money.”

- Evans RG.

Manufacturing consensus, marketing truth: Guidelines for

economic evaluation. Ann Intern Med 1995;123:59-60.

18

19

Underutilization of Recommended Services:How Effective Can Pharmaceuticals Be in Usual Community Practice?

McGlynn E, Asch AM, Adams J, et al. The Quality

of Health Care Delivered to Adults in the United

States. New England Journal of Medicine

2003;348:2536-2645.

20

21

22

Concentration of Health Spending in the Total

U.S. and Family Populations, 2002*

Source: Kaiser Family Foundation. Trends and Indicators, 2005 Update, Exhibit 1.11.

Geographic Variation in Medicare Drug Spending*

Zhang Y, Baicker K and Newhouse JP. NEJM 2010;

Definition:

Comparative Effectiveness Research (CER)

CER is the generation and synthesis of evidence that compares

the benefits and harms of alternative methods to prevent,

diagnose, treat, and monitor a clinical condition or to improve

the delivery of care.

The purpose of CER is to assist consumers, clinicians,

purchasers, and policy makers to make informed decisions that

will improve health care at both the individual and population

levels.

24Source: Initial National Priorities for Comparative Effectiveness Research . Institute of Medicine Report Brief June 2009. Retrieved from

http://www.iom.edu/~/media/Files/Report%20Files/2009/ComparativeEffectivenessResearchPriorities/CER%20report%20brief%2008-13-09.ashx

25

JAMA 2002;288:2981-2997

Comparative Effectiveness: What Can It Do?

26

MMA, P.L.

108-173

ARRA, P.L.

111-5

Senate

Finance

Committee

Senate

HELP

Committee

House of

RepresentativesGovernment

Agencies

PPACA

2010

CER under

AHRQ

CER under

Federal

Coordinating

Council

Creates a new

Public-Private

entity

CER under

AHRQ

CER under

AHRQ

Existing

policies &

Process for

COI

New Public-

Private

entity (Patient-

Centered

Outcomes

Research

Institute)

IOM

Determine

Funding

Priorities

Governing

Board (15)

3 from industry

Peer Review

decides

actions

Governing

Board (19)

incl: private

payers,

pharm’l,

device, &

diagnostic

manufacturer

s & developers

$1.1B over 2

years for CER

Methodology Cte

Industry members

27

Comparative Effectiveness Research

& US Health Care Reform

Selker, HP and Wood, AJJ. Industry Influence on Comparative Effectiveness Research Funded Through Healthcare

Reform. NEJM 2009;361(27):2595-2597. ; and P.L. 111-148

Patient Centered Outcomes Research Institute (PCORI http://www.pcori.org/aboutus.html)

The purpose of the Institute is to assist patients, clinicians,

purchasers, and policy-makers in making informed health

decisions by advancing the quality and relevance of evidence

… through research and evidence synthesis that considers

variations in patient subpopulations,

…

identifying effective strategies for organizing, financing, or

delivering public health services in real world community

settings, including comparing State and local health

department structures and systems in terms of effectiveness and

cost.

28

*Source: Patient Protection and Affordable Care Act of 2010. PL 111-148, sec. 6301

http://www.gpo.gov/fdsys/pkg/PLAW-111publ148/pdf/PLAW-111publ148.pdf

Patient Centered Outcomes Research Institute (PCORI http://www.pcori.org/aboutus.html)

The purpose of the Institute is the dissemination of

research findings with respect to the relative health

outcomes, clinical effectiveness, and appropriateness

of the medical treatments, services, …

29

*Source: Patient Protection and Affordable Care Act of 2010. PL 111-148, sec. 6301

http://www.gpo.gov/fdsys/pkg/PLAW-111publ148/pdf/PLAW-111publ148.pdf

Patient Centered Outcomes Research Institute (PCORI http://www.pcori.org/aboutus.html)

Prohibited:

Insurance coverage decisions/ recommendations

Producing clinical practice guidelines

Developing or using metrics that provide a value threshold (QALYs)

Principles

Transparency

Public-Private entity, i.e., non-governmental

21 member board: broad stakeholder representation

Stable funding

Priority setting

Methodology committee

30

*Sources: Patient Protection and Affordable Care Act of 2010. PL 111-148, sec. 6301

http://www.gpo.gov/fdsys/pkg/PLAW-111publ148/pdf/PLAW-111publ148.pdf ;

Garber AM & Sox HC. The Role of Costs In Comparative Effectiveness Research. Health Affairs 2010;29(10):1805-12811.

Part II

1) The evolution of comparative effectiveness research

& the Patient Centered Outcomes Research

Institute’s (PCORI's) potential impact on medical

publications,

2) A vision for the future of medical publications in a

market-driven health care system, and

3) Core competencies for medical publications

professionals in this new world.

31

Four Principles of Comparative

Effectiveness Analyses*

1) “Real-world” setting: usual community practice, with

placebo comparators generally not an option

2) Representative populations: less restrictive than RCTs

3) Personalized health care: Patient-specific & preferences

4) Full information: multiple outcomes, particularly patient

concerns such as AEs, side effect risks

32

* Garber AM & Sox HC. The Role of Costs In Comparative Effectiveness Research. Health Affairs 2010;29(10):1805-12811.

ePublishing & Beyond

• Comparative effectiveness results (PCORI) will support

improved private sector cost effectiveness analysis (Garber &

Sox,2010)

Futuristic, but is the future now?

• Actual real-time global readership

• Rapid TAT, with no page limit(s)

• Peer Reviewed Communications > text alone

• Post-publication review (Rennie & Luft, 2000)

• Social media (pending FDA guidance)

• Comprehensive publication strategies

• Richer metrics than Impact Factor alone

33Rennie D, Luft HS. Pharmacoeconomic analyses: making them transparent, making them credible. JAMA. 2000 Apr

26;283(16):2158-60.

Part III

1) The evolution of comparative effectiveness research

& the Patient Centered Outcomes Research

Institute’s (PCORI's) potential impact on medical

publications,

2) A vision for the future of medical publications in a

market-driven health care system, and

(3) Core competencies for medical publications

professionals in this new world.

34

Medical Publication Professionals’ Competencies:

More Than Health Economics

Core Competencies

• RCT and pragmatic clinical trials

• Phase IV (post-marketing) methodologies

• Pharmacovigilance

• Health economics’ core concepts

• Innovative data visualization skills

(http://www.edwardtufte.com/tufte/)

• Quantitative reasoning & modeling concepts

• Personalized medicine

35

Professional Practice

• Good Research for Comparative Effectiveness (GRACE) Principles 1

• Systematic reviews per PRISMA (http://www.prisma-statement.org/) 2

• Process and hierarchy of evidence-based practice

• Guidelines for Good Pharmacoepidemiologic Practices 3

• ICMJE Uniform Disclosure Form for Potential COI (JAMA 2010)

• Cochrane Risk Bias Tool (Lundh & Gøtzsche)4

• ISPOR’s Health Economic Publication Guidelines Task Force

(http://www.ispor.org/taskforces/EconomicPubGuidelines.asp)

•

36

Medical Publication Professionals’ Competencies:

More Than Health Economics

1 Dreyer N, et al. GRACE principles. AJMC 2010;16:467-471. ; 2 Lyles A. Systematic Reviews:

When the Published Literature is the Data. Clinical Therapeutics 2010; 32(10):1754-1755.;

3Pharmacoepidemiology Drug Safety 2008(17):200-208;4. Lundh A & Gøtzsche PC.

Recommendations by Cochrane Review Groups for assessment of the risk of bias in studies.

BMC Med Res Methodol. 2008; 8: 22 doi: 10.1186/1471-2288-8-22.

Medical Publication Professionals’ Competencies:

More Than Health Economics

Critical Competitive Edge: General systems thinking that

integrates clinical, economic, humanistic and policy

considerations for feasible recommendations.

37

Survival and Success

“In our view, the companies that will survive and

thrive in this new environment will be those that

embrace comparative effectiveness research (CER)

as the next logical step in the progression of requiring

evidence and recognize it as a necessary input for a

value-driven healthcare system.”*

38

Berger ML, Grainger D. Comparative effectiveness research: the view from a pharmaceutical company. Pharmacoeconomics.

2010;28(10):915-922. cited in Iglehart JK. The Political Fight Over Comparative Effectiveness Research.

Health Affairs 2010;10:1757-1760.

Welcome to….

The 7th Annual Meeting of

ISMPP

Anticipating Change in Medical

Publications:

Leading Now for the Future

SUPPLEMENTAL MATERIAL

Filibuster & Cloture:United States Senate

Filibuster: Informal term … any attempt to block or delay Senate

action on a bill … by offering numerous procedural motions, or

by any other delaying or obstructive actions.

Cloture: The only procedure by which the Senate can vote to place a

time limit on consideration of a bill … and overcome a filibuster.

Under the cloture rule (Rule XXII), the Senate may limit consideration of a

pending matter to 30 additional hours, but only by vote of three-fifths of the

full Senate, normally 60 votes.

41

Sources: http://www.senate.gov/reference/glossary_term/filibuster.htm

http://www.senate.gov/reference/glossary_term/cloture.htm

42

0

20

40

60

80

100

120

140

160

Cou

nt

Senate Action on Cloture Motions(Source: United States Senate http://www.senate.gov/pagelayout/reference/cloture_motions/clotureCounts.htm )

Motions Filed

Votes on Cloture

Cloture Invoked

Patient Centered Outcomes Research

Institute (PCORI http://www.pcori.org/aboutus.html)

PCORI Governing Board http://www.gao.gov/about/hcac/patientcentered_outcomes.html

43

44

Outcomes Variation from Efficacy to Effectiveness of Pharmacotherapy

(OVERxTx):

A Post-Marketing Systems Perspective

Patient / Condition

Physician / Provider

Efficacy Potential

Outcomes

Administrative

Unit(s)

Realized

Outcomes

Hetero

gen

eity

Product(s)

[EB]

Service(s)

[EB]

Adherence

(regimen)

Adherence

(guideline)

Cost Responsibilities

Qualifying Criteria

Data

Bo

th

C. Alan Lyles 2008

PublicPrivate

For-Profit

Private

Non-Profit

A diagnostic search for gaps, discontinuities, conflicts and/or redundancies.(Effectiveness)

45

November 15th, 1941

On the cusp of change

46

Health

Plans

*Source: CMS, Office of Research, Development & Information

PBM

Mail Order

Pharmacy

Pharmaceutical

Manufacturer

WholesalerRetail Pharmacy

Consumer

Shared Discounts/

Rebates

Admin Fees

Ingredient

Cost

Discounts/

Rebates

Co-PayCo-Pay

Ingredient

Cost

Dispensing

Fee

Cost of

Drug +

Wholesaler

Margin

Cost of

Drug

Money Flow in The Pharmaceutical Distribution Chain*

47

Cost of Drug Development

Cost ~ $500 - $800 million to develop a drug

Why is it so expensive ?

• Average length of time is 15 years

• Cost of capital for R&D

• Companies are exposed to a lot of risks and uncertainties

Tufts Center for the Study of Drug Development:

http://csdd.tufts.edu/InfoServices/Publications.asp

48

A Private Sector, Demand Driven Approach

Annual Meeting, San Diego October 2000

Journal of Managed Care Pharmacy July/August 2001;7(4):272-282.Version 3.0 http://www.amcp.org/format/pub.pdf

Public-Private Arrangements

• “Any arrangement between government and the private sector

in which partially or traditionally public activities are

performed by the private sector.” (Savas, 2004)

• Distinguished from Outsourcing and Privatization, Public

Private Partnerships are long term

• Accountability implications (Forrer, et al. 2010)

49

Savas, E.S. (2000). Privatization and Public-Private Partnerships. New York: Chatham House.

Forerr, J, et al. (2010). Public-Private Partnerships & the Public Accountability Question.

PAR 70(3):475-484.

Public-Private Partnerships in Healthcare:

Complementary Circumstances

Public Sector

+ Safety net

+ Standards & Enforcement

+ Collective societal goods

+ Managing COI

- Innovation

- Payment Systems

- Productivity

- Options > basic insurance

- Limited Funds

Private Sector

+ Innovation

+ Payment Systems

+ Efficiency

+ Insurance options

- Medically Uninsurable

- Setting standards

- Enforcement / Accountability

- Limited Funds

Source: Morlock LL, Waters HR, Lyles A, Ozsari H, and Aktulay G. Health Care Reform in Turkey – Charting the Way Forward. The Turkish Industrialists‘ and Businessmen‘s Association (TÜSIAD). August 31, 2004.

51

Comparative Effectiveness:

MMA (2003) Section 1013

• authorizes the Agency for Healthcare Research and Quality (AHRQ) to conduct research, demonstrations, and evaluations designed to improve the quality, effectiveness, and efficiency of Medicare, Medicaid, and the State Children's Health Insurance Program (SCHIP)

• The goals of Section 1013 are:

To develop valid evidence about the comparative effectiveness of different treatments and appropriate clinical approaches to difficult health problems.

To make the information easily accessible and understandable to decisionmakers.

Source: Agency for Healthcare Research and Quality (AHRQ)

http://effectivehealthcare.ahrq.gov/aboutUs/faq.cfm

Welcome to….

The 7th Annual Meeting of

ISMPP

Anticipating Change in Medical

Publications:

Leading Now for the Future